Last week, Othera Pharmaceuticals, Inc., a specialty pharmaceutical company focused on treatments for ophthalmic diseases, announced positive interim data results from its Phase 2 trial of OT-551 in treating geographic atrophy (GA), an advanced form of dry age-related macular degeneration (AMD) for which there is no FDA-approved treatment. The 12-month findings from the 2-year OMEGA trial suggest an emerging trend for reducing moderate vision loss (i.e., 15 letters or more on the ETDRS chart) in patients with GA who were treated with OT-551 compared with placebo. This numeric trend was more pronounced in subgroups based on GA characteristics or level of visual acuity at baseline.

According to Othera’s press release, OT-551 is a topically-dosed, patented small molecule that acts on oxidative stress and disease-induced inflammation. Othera observed that a number of scientific publications from leading researchers in ophthalmology have linked both oxidative stress and inflammation to the progression of GA and the ensuing vision loss in patients. OT-551 has demonstrated a dose-dependent protective effect on photoreceptor activity in an animal model of AMD, and has been shown to reach the back of the eye after topical dosing in multiple species. This profile supports the rationale for studying the drug in patients with degenerative retinal conditions, such as GA. OT-551 is the first eye drop to ever be tested in a clinical trial as a treatment for dry AMD.

A topical application that will slow down the second most devastating form of macular degeneration is very encouraging. Geographic atrophy (GA) causes vision loss by damaging large confluent areas/patches of RPE. Vision loss is severe when these “areas” involved the macular. This is in contrast to the “wet” form which involves choroidal neovascularization.

In this age of intravitreal injections and drug delivery, it makes me wonder how effective this might be in the future when the drug is more directly delivered to the retina. The socio-economic implications are very exciting. Therapy for the exudative form has been established. Perhaps we are closing in on treatments for the non-exudative forms as well.